Craig Mayer, Nick WIlliams, Vojtech Huser
Lister Hill National Center for Biomedical Communications, National Library of Medicine, NIH, Bethesda, MD

See the full regCTG repository at https://github.com/lhncbc/CRI/tree/master/regCTG

To return to the list of conditions and view other reports go to https://lhncbc.github.io/CRI/regCTG/regCTG-condition_report_links.html


Study Counts

Type Study_count
Total_Studies 116
interventional 68
Observational 43
Registry 5

Studies over Time by Start Date

```

Interventional Trials

Intervention Type

intervention_type Study_Count
Drug 31
Biological 18
Genetic 4
Drug|placebo 3
Other 3
Biological|Drug 2
Device|Procedure 2
Biological|placebo 1
Device 1
Drug|Other|placebo 1
Other|Procedure 1
Procedure 1

Count of Studies by Intervention (normalized and mapped)

Interventions over Time

Yearly studies for each intervention by start date

Studies by Country

Country Study_Count
United States 16
Canada 3
France 2
Germany 2
NA 2
United States|Australia|Austria|Belgium|Canada|Germany|Ireland|Italy|Netherlands|Norway|Poland|Spain|Switzerland|United Kingdom 2
United States|Canada|Czech Republic|Hungary|Poland|United Kingdom 2
United States|France|Poland|Russian Federation|United Kingdom 2
Australia 1
Australia|Brazil 1
Australia|Canada|Czechia|Germany|Netherlands|Norway|Slovenia|Spain|United Kingdom 1
Australia|United Kingdom 1
Czech Republic|Estonia|Poland|Slovakia 1
Czechia 1
Denmark 1
France|United Kingdom 1
Italy 1
Italy|Norway|United Kingdom 1
Japan 1
Korea, Republic of 1
Netherlands 1
Spain 1
Switzerland 1
United Kingdom 1
United States|Argentina|Australia|Austria|Belgium|Brazil|Canada|Denmark|Egypt|France|Italy|Japan|Spain|Turkey|United Kingdom 1
United States|Argentina|Australia|Belgium|Brazil|Canada|Czechia|Finland|France|Germany|Hungary|Italy|Netherlands|Norway|Paraguay|Slovenia|Spain|Switzerland|Turkey|United Kingdom 1
United States|Argentina|Australia|Brazil|Canada|Denmark|Egypt|France|Italy|Poland|Spain|Turkey|United Kingdom 1
United States|Argentina|Brazil|Canada|Czech Republic|Netherlands|Norway|Poland|United Kingdom 1
United States|Australia|Austria|Belgium|Brazil|Denmark|France|Italy|Japan|United Kingdom 1
United States|Australia|Belgium|Canada|Netherlands 1
United States|Australia|Brazil|Canada|France|United Kingdom 1
United States|Australia|Canada 1
United States|Australia|Canada|Czechia|Hungary|Netherlands|Norway|Slovenia|Spain|United Kingdom 1
United States|Australia|Czechia|Finland|Paraguay|Poland|Slovenia|United Kingdom 1
United States|Australia|Czechia|Finland|Poland|Slovenia|United Kingdom 1
United States|Australia|Paraguay|Serbia|Spain|United Kingdom 1
United States|Austria|United Kingdom 1
United States|Belgium|Canada|Czechia|Denmark|Italy|Netherlands|Norway|Spain|Taiwan|Turkey|United Kingdom 1
United States|Belgium|Czechia|Denmark|Italy|Norway|United Kingdom 1
United States|Canada 1
United States|France|Netherlands|Puerto Rico|United Kingdom 1
United States|France|Poland|United Kingdom 1
United States|Paraguay|Spain|United Kingdom 1
United States|Spain 1
United States|Spain|United Kingdom 1

Sites per Study

Site_count Study_Count
1 22
2 4
3 3
4 2
5 5
6 3
7 5
8 2
9 1
10 1
11 1
12 4
14 3
20 2
21 1
24 1
25 2
26 1
28 1
29 1
51 1
58 2

Phase

Phase Study_Count
Phase 3 17
Phase 2 16
N/A 12
Phase 1 10
Phase 4 8
Phase 1/Phase 2 5

Number of Arms

Number_of_Arms Count_of_Studies
1 43
2 13
3 3
4 1
5 2
6 1
NA 5

Enrollment Metrics by Phase

Measure N.A Phase.1 Phase.1.Phase.2 Phase.2 Phase.3 Phase.4
Min. 12.0000 3.0 12 3.000 20 6.00
1st Qu. 20.0000 6.0 15 8.750 31 18.75
Median 51.0000 11.0 15 14.000 58 26.50
Mean 141.0833 13.9 18 13.625 64 35.25
3rd Qu. 109.2500 18.5 18 17.750 78 46.00
Max. 1000.0000 32.0 30 25.000 171 82.00

Trial Group Type

group_type Group_Count
Experimental 79
Active Comparator 8
Other 6
NA 5
Placebo Comparator 4
No Intervention 1

Intervention Model

intervention_model Study_Count
Single Group Assignment 44
Parallel Assignment 16
NA 4
Crossover Assignment 2
Sequential Assignment 2

Primary Purpose

primary_purpose Study_Count
Treatment 55
Diagnostic 7
Basic Science 2
NA 2
Other 1
Supportive Care 1

Observational Studies

Studies by Country

Country Study_Count
United States 13
France 4
Germany 4
NA 3
Denmark 2
Korea, Republic of 2
Argentina|Belgium|Croatia|Czechia|Denmark|France|Germany|United Kingdom 1
Austria 1
Austria|Belgium|Croatia|Finland|France|Georgia|Germany|Ireland|Italy|Malta|Poland|Portugal|Spain|United Kingdom 1
Austria|Croatia|France|Georgia|Germany|Poland|Portugal 1
Austria|Germany 1
Belgium 1
Belgium|Czech Republic|Denmark|Finland|Germany|Sweden 1
Czechia 1
Germany|India|Sri Lanka 1
Germany|Poland 1
Italy 1
Japan 1
Switzerland 1
United Kingdom 1
United States|Slovenia 1

Sites per Study

Site_count Study_Count
1 30
2 3
3 1
6 1
9 2
11 1
14 1
16 1
20 1
22 1
45 1

Enrollment Metrics

Measure Observational
Min 4.0000
1st Qu 32.5000
Median 100.0000
Mean 526.1628
3rd Qu 520.0000
Max 5000.0000

Observation Model

observational_model Study_Count
Cohort 26
NA 9
Case-Only 6
Case Control 1
Other 1

Time Perspective

time_perspective Study_Count
Prospective 27
NA 6
Cross-Sectional 4
Retrospective 4
Other 2

Registries

Studies by Country

Country Study_Count
Canada 2
United States 2
United States|Argentina|Australia|Belgium|Brazil|Bulgaria|Canada|Chile|China|Colombia|Croatia|Czechia|Denmark|Estonia|Finland|France|Germany|Hong Kong|Hungary|India|Indonesia|Italy|Japan|Korea, Republic of|Kuwait|Lithuania|Malaysia|Netherlands|Norway|Peru|Philippines|Poland|Portugal|Romania|Russian Federation|Singapore|Slovakia|Spain|Sweden|Taiwan|Thailand|United Kingdom 1

Sites per Study

Site_count Study_Count
1 3
5 1
219 1

Enrollment Metrics

Measure Registries
Min 8.0
1st Qu 600.0
Median 4000.0
Mean 3721.6
3rd Qu 5000.0
Max 9000.0

Registry Model

observational_model Study_Count
Cohort 2
Other 2
Case-Control 1

Time Perspective

time_perspective Study_Count
Prospective 3
Other 2

Follow-up

target_duration Study_Count
1 Day 1
10 Years 1
2 Years 1
25 Years 1
52 Weeks 1

Overview of Studies

Interventional Trials

#If less then 500 trials

nct_id brief_title link overall_status source primary_completion_date
NCT03305250 Arrhythmia Burden, Risk of Sudden Cardiac Death and Stroke in Patients With Fabry Disease https://ClinicalTrials.gov/show/NCT03305250 Recruiting University Hospital Birmingham NHS Foundation Trust 2022-01-31
NCT03180840 Study of the Safety, Efficacy, & PK of Pegunigalsidase Alfa (PRX-102) 2 mg/kg IV Administered Every 4 Weeks in Fabry Disease Patients https://ClinicalTrials.gov/show/NCT03180840 Active, not recruiting Protalix 2020-07-31
NCT04455230 A Long Term Follow-Up Study of Fabry Disease Subjects Treated With FLT190 https://ClinicalTrials.gov/show/NCT04455230 Recruiting Freeline Therapeutics 2026-12-31
NCT03018730 Safety and Efficacy of PRX 102 in Patients With Fabry Disease Currently Treated With REPLAGAL® (Agalsidase Alfa) https://ClinicalTrials.gov/show/NCT03018730 Active, not recruiting Protalix 2020-01-31
NCT02995993 Pharmacokinetics, Pharmacodynamics, and Safety of Moss-aGalactosidase A in Patients With Fabry Disease https://ClinicalTrials.gov/show/NCT02995993 Completed Greenovation Biotech GmbH 2017-10-09
NCT02985710 Assessment of Small Fiber Neuropathy in Rare Diseases Using Sudoscan https://ClinicalTrials.gov/show/NCT02985710 Recruiting Massachusetts General Hospital 2019-02-28
NCT02930655 A Study to Assess the Safety and Tolerability of Lucerastat in Subjects With Fabry Disease https://ClinicalTrials.gov/show/NCT02930655 Completed Idorsia Pharmaceuticals Ltd. 2016-02-01
NCT02800070 Autologous Stem Cell Transplantation of Cells Engineered to Express Alpha-Galactosidase A in Patients With Fabry Disease https://ClinicalTrials.gov/show/NCT02800070 Active, not recruiting University Health Network, Toronto 2024-02-29
NCT02795676 Study of the Safety and Efficacy of PRX-102 Compared to Agalsidase Beta on Renal Function https://ClinicalTrials.gov/show/NCT02795676 Active, not recruiting Protalix 2021-10-31
NCT02489344 Evaluation of the Long-term Safety, Pharmacodynamics, and Exploratory Efficacy of GZ/SAR402671 in Treatment-Naïve Adult Male Patients With Fabry Disease https://ClinicalTrials.gov/show/NCT02489344 Completed Sanofi 2018-11-20
NCT02450604 Prevalence of Fabry’s Disease in a Population of Patients With Chronic Pain https://ClinicalTrials.gov/show/NCT02450604 Recruiting University Hospital, Bordeaux 2020-05-05
NCT02228460 Evaluate the Safety, Pharmacodynamics, Pharmacokinetics, and Exploratory Efficacy of GZ/SAR402671 in Treatment-naïve Adult Male Patients With Fabry Disease https://ClinicalTrials.gov/show/NCT02228460 Completed Sanofi 2016-09-30
NCT02194985 Open-Label Extension Study of the Long-Term Effects of Migalastat HCL in Patients With Fabry Disease https://ClinicalTrials.gov/show/NCT02194985 Active, not recruiting Amicus Therapeutics 2020-12-31
NCT02090608 Paricalcitol in Fabry Disease https://ClinicalTrials.gov/show/NCT02090608 Completed Federico II University 2013-12-31
NCT02082327 A Phase 1 Study To Evaluate the Safety of Migalastat Hydrochloride Given Intravenously to Healthy Volunteers https://ClinicalTrials.gov/show/NCT02082327 Completed Amicus Therapeutics 2014-06-30
NCT01304277 This Study is Designed to Evaluate PD/PK and Safety of Replagal Manufactured by Two Different Processes. https://ClinicalTrials.gov/show/NCT01304277 Completed Shire 2012-12-31
NCT01298141 A Multicenter Open-Label Treatment Protocol to Observe the Safety of Replagal (Agalsidase Alfa) Enzyme Replacement Therapy in Canadian Patients With Fabry Disease https://ClinicalTrials.gov/show/NCT01298141 Completed Shire 2017-09-25
NCT01997489 Ophthalmic Findings During 10-year Enzyme Substitution of Danish Fabry Patients. https://ClinicalTrials.gov/show/NCT01997489 Completed Rigshospitalet, Denmark 2013-09-30
NCT01981720 Extension Study of PRX-102 for up to 60 Months https://ClinicalTrials.gov/show/NCT01981720 Active, not recruiting Protalix 2021-03-31
NCT01947634 Sleepiness and Sleep-disordered Breathing in Fabry Disease. A Prospective Cohort Study. https://ClinicalTrials.gov/show/NCT01947634 Completed University of Zurich 2015-01-31
NCT01853852 A Phase I, Randomized, Single-Blind, Four-Period Cross-Over, Placebo-Controlled, Dose-Escalation Study to Evaluate the Safety and Pharmacokinetics of Single Oral Doses of GR181413A/AT1001 in Healthy Japanese Subjects https://ClinicalTrials.gov/show/NCT01853852 Completed Amicus Therapeutics 2011-12-31
NCT01730482 A Study to Assess the Absorption, Metabolism and Excretion of Migalastat Hydrochloride (AT1001-014) https://ClinicalTrials.gov/show/NCT01730482 Completed Amicus Therapeutics 2011-09-30
NCT01730469 Safety and Pharmacokinetics of AT1001 (Migalastat HCl) in Healthy Subjects and Subjects With Impaired Renal Function https://ClinicalTrials.gov/show/NCT01730469 Completed Amicus Therapeutics 2012-04-30
NCT01678898 Dose-ranging Study of PRX-102 in Adult Fabry Disease Patients https://ClinicalTrials.gov/show/NCT01678898 Completed Protalix 2016-03-06
NCT01653444 Evaluate the Safety and Exploratory Efficacy of GC1119 https://ClinicalTrials.gov/show/NCT01653444 Completed Green Cross Corporation 2014-05-31
NCT01650779 A Study Evaluating Glycosphingolipid Clearance in Patients Treated With Agalsidase Alfa Who Switch to Agalsidase Beta https://ClinicalTrials.gov/show/NCT01650779 Completed Sanofi 2013-03-31
NCT04002531 A One Visit Follow Up of Adults With Fabry Disease Who Started Long-term Enzyme Replacement Therapy As Children https://ClinicalTrials.gov/show/NCT04002531 Enrolling by invitation Baylor Research Institute 2019-12-31
NCT01489995 Migalastat Food Effect Study https://ClinicalTrials.gov/show/NCT01489995 Completed Amicus Therapeutics 2011-12-31
NCT01363492 Safety Study of Replagal® Therapy in Children With Fabry Disease https://ClinicalTrials.gov/show/NCT01363492 Completed Shire 2013-05-31
NCT04143958 To Assess the Glycosphingolipid Clearance and Clinical Effects of Switching to Agalsidase Beta (Fabrazyme) Versus Continuing on Agalsidase Alfa (Replagal) in Male Patients With Classic Fabry Disease https://ClinicalTrials.gov/show/NCT04143958 Recruiting Sanofi 2023-11-30
NCT04073888 Study of the Spermatic Characteristics of Patients With Fabry Disease https://ClinicalTrials.gov/show/NCT04073888 Completed University Hospital, Bordeaux 2013-10-18
NCT04050137 Therapeutic Exercise to Treat Neuropathic Pain https://ClinicalTrials.gov/show/NCT04050137 Active, not recruiting Universidad de Zaragoza 2020-12-30
NCT04049760 Safety, Pharmacodynamics, and Efficacy of Migalastat in Pediatric Subjects (Aged >12 Years) With Fabry Disease https://ClinicalTrials.gov/show/NCT04049760 Recruiting Amicus Therapeutics 2022-06-01
NCT04046224 Dose-Ranging Study of ST-920, a rAAV2/6 Human Alpha Galactosidase A Gene Therapy in Subjects With Fabry Disease https://ClinicalTrials.gov/show/NCT04046224 Recruiting Sangamo Therapeutics 2021-09-28
NCT04040049 A Fabry Disease Gene Therapy Study https://ClinicalTrials.gov/show/NCT04040049 Recruiting Freeline Therapeutics 2021-12-31
NCT03737214 A Study to Evaluate the Long-term Safety and Tolerability of Lucerastat in Adult Subjects With Fabry Disease https://ClinicalTrials.gov/show/NCT03737214 Recruiting Idorsia Pharmaceuticals Ltd. 2022-05-22
NCT03678324 Pre-Clinical White Matter Changes and Associated Connectivity Effects in Fabry Disease https://ClinicalTrials.gov/show/NCT03678324 Recruiting University of Utah 2022-07-30
NCT03614234 Open Label Extension of 2 mg/kg Pegunigalsidase Alfa (PRX-102) Every 4 Weeks in Adult Fabry Disease Patients https://ClinicalTrials.gov/show/NCT03614234 Enrolling by invitation Protalix 2023-01-31
NCT03566017 Extension Study of 1 mg/kg Pegunigalsidase Alfa in Patients With Fabry Disease https://ClinicalTrials.gov/show/NCT03566017 Enrolling by invitation Protalix 2022-09-30
NCT03500094 Safety, Pharmacokinetics, Pharmacodynamics, and Efficacy of Migalastat in Pediatric Subjects (Aged 12 to <18 Years) https://ClinicalTrials.gov/show/NCT03500094 Active, not recruiting Amicus Therapeutics 2021-03-01
NCT03454893 Open-Label, Study Of Efficacy and Safety Of AVR-RD-01 for Treatment -Naive Subjects With Classic Fabry Disease https://ClinicalTrials.gov/show/NCT03454893 Recruiting AvroBio 2021-07-31
NCT03425539 Efficacy and Safety of Lucerastat Oral Monotherapy in Adult Subjects With Fabry Disease https://ClinicalTrials.gov/show/NCT03425539 Recruiting Idorsia Pharmaceuticals Ltd. 2021-05-15
NCT01218659 Study to Compare the Efficacy and Safety of Oral AT1001 and Enzyme Replacement Therapy in Patients With Fabry Disease https://ClinicalTrials.gov/show/NCT01218659 Completed Amicus Therapeutics 2014-05-27
NCT01196871 Drug-Drug Interaction Study Between AT1001 (Migalastat Hydrochloride) and Agalsidase in Participants With Fabry Disease https://ClinicalTrials.gov/show/NCT01196871 Completed Amicus Therapeutics 2012-10-09
NCT01178164 Prevalence of Fabry’s Disease in a Population of Patients With Chronic Pains https://ClinicalTrials.gov/show/NCT01178164 Completed University Hospital, Bordeaux 2012-09-30
NCT01124643 Extension Study of TKT028 Evaluating Safety and Clinical Outcomes of Replagal® in Adult Patients With Fabry Disease https://ClinicalTrials.gov/show/NCT01124643 Completed Shire 2013-07-08
NCT00925301 Study of the Effects of Oral AT1001 (Migalastat Hydrochloride) in Patients With Fabry Disease https://ClinicalTrials.gov/show/NCT00925301 Completed Amicus Therapeutics 2012-06-12
NCT00864851 Safety and Efficacy Study of Several Replagal Dosing Regimens on Cardiac Function in Adults With Fabry Disease https://ClinicalTrials.gov/show/NCT00864851 Completed Shire 2012-06-01
NCT00230607 Study of the Effects of Fabrazyme Treatment on Lactation and Infants https://ClinicalTrials.gov/show/NCT00230607 Recruiting Sanofi 2020-07-31
NCT02798458 Evaluation of the Gastrointestinal Manifestation of Fabry’s Disease https://ClinicalTrials.gov/show/NCT02798458 Recruiting Massachusetts General Hospital 2020-07-31
NCT00701415 A Study of Two Fabrazyme (Agalsidase Beta) Dosing Regimens in Treatment-naïve, Male Pediatric Patients Without Severe Symptoms https://ClinicalTrials.gov/show/NCT00701415 Completed Sanofi 2015-06-30
NCT00357786 An Open-Label Maintenance Study of the Enzyme Replacement Therapy Replagal in Patients With Fabry Disease https://ClinicalTrials.gov/show/NCT00357786 Completed National Institutes of Health Clinical Center (CC) 2008-12-31
NCT00304512 A 12-Week Safety and Pharmacodynamic Study of AT1001 (Migalastat Hydrochloride) in Female Participants With Fabry Disease https://ClinicalTrials.gov/show/NCT00304512 Completed Amicus Therapeutics 2008-05-09
NCT00283959 A 12-Week Safety and Pharmacodynamic Study of AT1001 (Migalastat Hydrochloride) in Participants With Fabry Disease https://ClinicalTrials.gov/show/NCT00283959 Completed Amicus Therapeutics 2008-05-08
NCT00283933 A 24-Week Safety and Pharmacodynamic Study of AT1001 (Migalastat Hydrochloride) in Participants With Fabry Disease https://ClinicalTrials.gov/show/NCT00283933 Completed Amicus Therapeutics 2008-03-12
NCT00214500 A Study of AT1001 (Migalastat Hydrochloride) in Participants With Fabry Disease https://ClinicalTrials.gov/show/NCT00214500 Completed Amicus Therapeutics 2008-01-29
NCT00196716 A Study of the Safety and Efficacy of Fabrazyme in Patients With Fabry Disease https://ClinicalTrials.gov/show/NCT00196716 Completed Sanofi 2006-04-30
NCT00140621 A Safety and Efficacy Study of Fabrazyme® Replacement Therapy in Patients With Cardiac Fabry Disease https://ClinicalTrials.gov/show/NCT00140621 Completed Sanofi 2012-08-31
NCT00097890 Replagal Enzyme Replacement Therapy for Adults With Fabry Disease https://ClinicalTrials.gov/show/NCT00097890 Completed National Institutes of Health Clinical Center (CC) NA
NCT00084084 Replagal Enzyme Replacement Therapy for Children With Fabry Disease https://ClinicalTrials.gov/show/NCT00084084 Completed Shire 2011-06-30
NCT00081497 A Study of the Safety and Efficacy of Fabrazyme in Patients With Fabry Disease https://ClinicalTrials.gov/show/NCT00081497 Completed Sanofi 2005-09-30
NCT00075244 Alternative Dosing and Regimen of Replagal to Treat Fabry Disease https://ClinicalTrials.gov/show/NCT00075244 Completed National Institutes of Health Clinical Center (CC) NA
NCT00074984 A Study of the Safety and Efficacy of Fabrazyme (Agalsidase Beta) as Compared to Placebo in Patients With Advanced Fabry Disease https://ClinicalTrials.gov/show/NCT00074984 Completed Sanofi 2004-01-31
NCT00074971 A Study of the Safety and Efficacy of Fabrazyme in Patients With Fabry Disease https://ClinicalTrials.gov/show/NCT00074971 Completed Sanofi NA
NCT00074958 A Study of Fabrazyme in Pediatric Patients With Fabry Disease https://ClinicalTrials.gov/show/NCT00074958 Completed Sanofi 2005-05-31
NCT00071877 An Open-Label Clinical Trial of Replagal Enzyme Therapy in Children Ages 7-17 Years With Fabry Disease https://ClinicalTrials.gov/show/NCT00071877 Completed National Institutes of Health Clinical Center (CC) NA
NCT00068107 Dosing Study of Replagal in Patients With Fabry Disease https://ClinicalTrials.gov/show/NCT00068107 Completed Baylor Research Institute 2013-12-31
NCT00048906 Alpha-Galactosidase A Replacement Therapy for Fabry Disease https://ClinicalTrials.gov/show/NCT00048906 Completed National Institutes of Health Clinical Center (CC) NA

Observational Studiess

#If less then 500 studies

nct_id brief_title link overall_status source primary_completion_date
NCT03362164 Evaluation of HEArt invoLvement in Patients With FABRY Disease https://ClinicalTrials.gov/show/NCT03362164 Recruiting Wuerzburg University Hospital 2032-03-31
NCT03230149 Prevalence and Characteristics of Fabry Disease (FD) in Patients With Stroke or Small Fiber Neuropathy https://ClinicalTrials.gov/show/NCT03230149 Recruiting Assistance Publique - Hôpitaux de Paris 2020-03-31
NCT03135197 German Observational Multicenter Study of Patients With Fabry Disease Under Chaperone Therapy With Migalastat-HCl. https://ClinicalTrials.gov/show/NCT03135197 Active, not recruiting University Hospital Muenster 2020-07-31
NCT02969200 Fabry: Renal Function During Long-term ERT by 51Cr-EDTA Clearance https://ClinicalTrials.gov/show/NCT02969200 Completed Rigshospitalet, Denmark 2016-09-30
NCT02908724 Cardiac Involvement in Adult Patients With Fabry Disease; Relation to Enzyme Replacement Therapy https://ClinicalTrials.gov/show/NCT02908724 Completed Rigshospitalet, Denmark 2014-11-30
NCT02778295 Biomarker for Patients With Fabry Disease (BioFabry) https://ClinicalTrials.gov/show/NCT02778295 Active, not recruiting Centogene AG Rostock 2021-06-30
NCT02719249 Fabry Disease Screening in ESRD Patients in West of France https://ClinicalTrials.gov/show/NCT02719249 Completed Rennes University Hospital 2018-06-30
NCT02469181 Impact of Enzyme Replacement Therapy on Cardiac Function in Patients With Fabry’s Cardiomyopathy (RECAFTURE Trial) https://ClinicalTrials.gov/show/NCT02469181 Recruiting Yonsei University 2020-10-31
NCT02152189 Screening for Fabry Disease in a Pediatric Population at Risk https://ClinicalTrials.gov/show/NCT02152189 Completed University Hospital Heidelberg 2017-05-31
NCT01295008 Androgenetic Alopecia in Fabry Disease https://ClinicalTrials.gov/show/NCT01295008 Completed Baylor Research Institute 2015-10-31
NCT01271699 Prevalence of Fabry Disease in a Defined Population at Risk - Patients Formerly Diagnosed With Multiple Sclerosis https://ClinicalTrials.gov/show/NCT01271699 Completed Centogene AG Rostock 2019-12-01
NCT01745185 Immune Response in Subjects With Fabry Disease Who Are Switching From Agalsidase Alfa to Agalsidase Beta https://ClinicalTrials.gov/show/NCT01745185 Completed O & O Alpan LLC 2015-04-07
NCT01632111 Pulmonary Involvement in Patients With Fabry Disease https://ClinicalTrials.gov/show/NCT01632111 Completed University of Zurich 2014-05-31
NCT01581424 Natural History and Structural Functional Relationships in Fabry Renal Disease Treatment Outcomes(Changes)in Fabry Renal Disease Study https://ClinicalTrials.gov/show/NCT01581424 Recruiting University of Minnesota 2020-07-31
NCT01355146 Home Therapy With Replagal in Fabry Disease https://ClinicalTrials.gov/show/NCT01355146 Completed Shire 2017-09-30
NCT04252066 A Global Prospective Observational Study of Women With Fabry Disease and Their Infants During Pregnancy and Breastfeeding https://ClinicalTrials.gov/show/NCT04252066 Recruiting Amicus Therapeutics 2029-08-31
NCT04043273 Treatment-related Benefit and Satisfaction in Fabry Patients. Insight in Patients Expectations and Preferences https://ClinicalTrials.gov/show/NCT04043273 Recruiting Amicus Therapeutics France SAS 2022-09-30
NCT03949920 A Study of Migalastat in Fabry Disease https://ClinicalTrials.gov/show/NCT03949920 Recruiting Manchester University NHS Foundation Trust 2021-12-01
NCT03886714 Screening for Fabry Disease in Renal Transplantation https://ClinicalTrials.gov/show/NCT03886714 Recruiting University Hospital, Montpellier 2021-03-25
NCT03838237 Effect of Migalastat on Cardiac Involvement in Fabry Disease https://ClinicalTrials.gov/show/NCT03838237 Active, not recruiting Ospedale San Donato 2020-12-31
NCT03683966 MigALastat Therapy Adherence Among FABRY Patients: A Prospective Multicentral Observational Study https://ClinicalTrials.gov/show/NCT03683966 Recruiting Wuerzburg University Hospital 2019-12-31
NCT03596398 Epidemiological Study of Fabry Disease in Taiwan Young Stroke Patients https://ClinicalTrials.gov/show/NCT03596398 Enrolling by invitation Chiayi Christian Hospital 2021-09-30
NCT01268241 The Efficacy and Safety of Switch Between Agalsidase Beta to Agalsidase Alfa for Enzyme Replacement in Patients With Anderson-Fabry Disease https://ClinicalTrials.gov/show/NCT01268241 Completed Centogene AG Rostock 2016-04-30
NCT01210196 Sophisticated Assessment of Disease Burden in Patients With Fabry Disease https://ClinicalTrials.gov/show/NCT01210196 Completed Shire 2013-09-30
NCT01198899 Belgian Screening Project for the Detection of Anderson-Fabry Disease in Hypertrophic Cardiomyopathy https://ClinicalTrials.gov/show/NCT01198899 Completed University Hospital, Ghent 2011-08-31
NCT01182818 Fabry and Stroke Epidemiological Protocol (FASEP): Risk Factors In Ischemic Stroke Patients With Fabry Disease https://ClinicalTrials.gov/show/NCT01182818 Completed University of Rostock 2012-04-30
NCT01165697 Establishment of Biomarkers for Fabry Disease https://ClinicalTrials.gov/show/NCT01165697 Completed Ohio State University 2014-07-31
NCT01019629 Fabry Screening Study https://ClinicalTrials.gov/show/NCT01019629 Completed Baylor Research Institute 2013-04-17
NCT03230591 Impact of Agalsidase Alfa Therapy on Cardiac funcTION in Patients With Fabry’s Cardiomyopathy https://ClinicalTrials.gov/show/NCT03230591 Recruiting Yonsei University 2019-06-30
NCT04184986 Screening of Fabry Disease in Patients With GI Symptoms https://ClinicalTrials.gov/show/NCT04184986 Recruiting General University Hospital, Prague 2020-09-01
NCT00728364 Anderson-Fabry Disease in Chronic Kidney Disease Patients Not on Renal Replacement Therapy https://ClinicalTrials.gov/show/NCT00728364 Completed Klinikum Wels-Grieskirchen 2011-08-31
NCT00446862 The Fabrazyme® and Arbs and ACE Inhibitor Treatment (FAACET) Study https://ClinicalTrials.gov/show/NCT00446862 Completed University of Alabama at Birmingham 2012-06-30
NCT00414583 Stroke in Young Fabry Patients (sifap1): Frequency of Fabry Disease in Young Stroke Patients https://ClinicalTrials.gov/show/NCT00414583 Completed Centogene AG Rostock 2010-01-31
NCT00413595 Stroke in Young Fabry Patients (sifap2): Characterization of the Stroke Rehabilitation https://ClinicalTrials.gov/show/NCT00413595 Completed Centogene AG Rostock 2019-12-01
NCT00343577 Antiproteinuric Agents and Fabry Disease https://ClinicalTrials.gov/show/NCT00343577 Completed University of Alabama at Birmingham NA
NCT00233870 A Long Term Safety and Efficacy Study of Fabrazyme Replacement Therapy in Japanese Patients With Fabry Disease. https://ClinicalTrials.gov/show/NCT00233870 Completed Sanofi 2011-03-31
NCT00168974 Neuropathic Pain and Fabry Disease https://ClinicalTrials.gov/show/NCT00168974 Completed Danish Pain Research Center NA
NCT00106912 Study to Collect Data on Fabry Disease Patients With Enhanceable Alpha-Galactosidase A Activity https://ClinicalTrials.gov/show/NCT00106912 Completed National Institutes of Health Clinical Center (CC) NA
NCT00005111 PET Scans in Normal Volunteers and Patients With Fabry Disease https://ClinicalTrials.gov/show/NCT00005111 Completed National Institutes of Health Clinical Center (CC) NA
NCT00001774 Vasodilation in Patients With Fabry’s Disease https://ClinicalTrials.gov/show/NCT00001774 Completed National Institutes of Health Clinical Center (CC) NA
NCT00030134 Data Collection in Women With Fabry Disease https://ClinicalTrials.gov/show/NCT00030134 Completed National Institutes of Health Clinical Center (CC) NA
NCT00055016 Registry of Fabry Disease - A Multicenter Observational Study https://ClinicalTrials.gov/show/NCT00055016 Completed National Institutes of Health Clinical Center (CC) NA
NCT00001491 Analysis of the Nervous System in Patients With Fabry’s Disease https://ClinicalTrials.gov/show/NCT00001491 Completed National Institutes of Health Clinical Center (CC) NA

Registries

#If less then 500 registries

nct_id brief_title link overall_status source primary_completion_date
NCT03321604 Kidney Information Network for Disease Research and Education https://ClinicalTrials.gov/show/NCT03321604 Recruiting Massachusetts General Hospital 2022-01-31
NCT03289065 Fabry Outcome Survey (FOS) https://ClinicalTrials.gov/show/NCT03289065 Recruiting Shire 2030-04-01
NCT02023086 Clinical Investigation on the Blood Oxygenation at the Optic Nerve Head in Fabry Patients https://ClinicalTrials.gov/show/NCT02023086 Completed Université de Montréal 2016-09-30
NCT00455104 Canadian Fabry Disease Initiative (CFDI) National Registry https://ClinicalTrials.gov/show/NCT00455104 Recruiting Canadian Fabry Research Consortium 2029-10-31
NCT00196742 Fabry Disease Registry & Pregnancy Sub-registry https://ClinicalTrials.gov/show/NCT00196742 Recruiting Sanofi 2034-01-31